NL-OMON54117招募中2 期
EPIK-P2: A Phase II double-blind study with an upfront, 16-week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS) - CBYL719F12201 (EPIK-P2)
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- ovartis
- 入组人数
- 14
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •1. Patients with diagnosis of PROS with symptomatic and /or progressive
- •overgrowth and at least one measurable PROS-related lesion confirmed by blinded
- •independent review committee (BIRC) assessment
- •2. Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in
- •local laboratories
- •3. A tissue sample (fresh or archival) must be available to be sent to a
- •Novartis-designated central laboratory. If archival tissue is not available,
- •collection of a fresh tissue biopsy is required for participants in Groups 1, 2
- •and 5, if it is not clinically contraindicated. For participants in Groups 3
- •and 4, a fresh tissue biopsy is not mandatory.
- •4. Karnofsky (in patients > 16 years old at study entry)/Lansky (<=16 yrs of age
- •at study entry) performance status index >=50
- •5. Adequate bone marrow and organ function including Fasting plasma glucose
- •(FPG) <= 140 mg/dL (7.7 mmol/L)* and Glycosylated hemoglobin (HbA1c) <= 6.5%
- •(both criteria have to be met) (as assessed by central laboratory for
- •eligibility within.
- •6. Presence of at least one PROS-related measurable lesion defined as a lesion
- •with longest diameter >=2 cm, when the volume can be accurately and reproducibly
- •measured by MRI, and associated with complaints, clinical symptoms or
- •functional limitations affecting the patient's everyday life. Measurability
- •must be confirmed by BIRC before randomization.
- •For the full inclusion criteria, please refer to Section 5.1. of protocol
排除标准
- •1. Participant with only isolated macrodactyly, epidermal nevus/nevi and
- •macroencephaly (the only clinical feature or a combination of any of three of
- •them), in absence of other PROS-related lesions at the time of informed consent.
- •2. Previous treatment with alpelisib and/or any other PI3K inhibitor(s) (except
- •treatment attempt, defined as the attempt to treat PROS with any of PI3K
- •inhibitors, with treatment duration less than 2 weeks and stopped at least 4
- •weeks prior to the first dose of study medication with alpelisib)
- •3. Radiation exposure for PROS treatment purpose within the previous 12 months
- •on those PROS areas which are expected to qualify for target lesions (except
- •lesion(s) progressing after completion of radiotherapy) at time of informed
- •4. Debulking or other major surgery performed within 3 months at time of
- •informed consent.
- •5. Clinically meaningful PROS-related thrombotic event (Grade 2 and more as per
- •CTCAE v.4.03) within 30 days before informed consent, and/or
- •sclerotherapy/embolization for vascular complications performed within 6 weeks
- •before informed consent. Participants (receiving anticoagulants for
- •PROS-related coagulopathy, primary or secondary prophylaxis of thrombosis may
- •be included in the study).
- •6. Participants with documented pneumonitis or interstitial lung disease at
- •time of informed consent.
- •7. History of acute pancreatitis within 1 year before informed consent or past
- •medical history of chronic pancreatitis at time of informed consent.
- •8. Participants with an established diagnosis of type I diabetes mellitus or
- •uncontrolled type II diabetes mellitus at time of informed consent.
- •9. Known history of seizure, or epilepsy, regardless of relatedness to PROS
- •sprectrum at time of informed consent, when epilepsy is not controlled and/or
- •the patient may not be switched to non-enzyme inducing antiepilectic drug(s) at
- •time of informed consent.
- •10. Participants with clinically significant worsening of the PROS-related
- •signs and symptoms (e.g. increase of D-dimers, worsening of underlying
- •pain, newly occurring swelling or redness) indicating an uncontrolled
- •condition during screening phase, particularly if systemic treatment with
- •any other inhibitor of the PI3K/AKT/mTOR pathway was stopped prior
- •to the start of the study treatment. This includes but is not limited to
- •hypercoagulability state in participants not receiving prophylactic
- •For the full inclusion criteria, please refer to Section 5.2 of protocol
研究者
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